The purpose of this study is to assess the dose dependent safety, tolerability and potential efficacy of QRX003 lotion in subjects with Netherton Syndrome (NS). This is a multi-center, randomized, vehicle-controlled, double-blind, parallel group comparison study of QRX003 lotion in adult subjects 18 years of age or older with NS. Approximately 18 subjects will be enrolled at approximately 7 sites. Subjects will be randomized (1:1:1) to treatment as follows:

1. QRX003 (dipalmitoyl hydroxyproline) lotion, 2% (Low dose)
2. QRX003 (dipalmitoyl hydroxyproline) lotion, 4% (High dose)
3. Vehicle lotion

Subjects will apply the assigned test article once daily in the morning to a designated Treatment Area for 12 weeks. Subjects will attend 5 clinic visits for up to 20 weeks.

This small stroke rehabilitation study will test the feasibility of a new method to personalize a home exercise program for arm/hand recovery. The research study will last ~8 weeks. Participants will be included if they are adults who have experienced a stroke that has caused one arm/hand to become weak, are able to come to the MUSC main campus 3 times for ~2hr arm movement evaluations, and are willing and able to engage in 60 minute telerehabilitation video visits with an occupational therapist 1-2 times per week for 6 weeks. We anticipate that the results of this study will enable occupational therapists and stroke survivors to, together, design home exercise programs that are meaningful, motivating and effective.

The study is designed to evaluate the effects of the combination of zibotentan and
dapagliflozin and dapagliflozin monotherapy versus placebo on the hepatic venous pressure gradient (HVPG) response in participants with cirrhosis with features of portal hypertension at 6 weeks of treatment.

Part A is to evaluate the absolute
change in HVPG at 6 weeks from baseline in participants treated with 2.5 mg zibotentan combined with 10 mg dapagliflozin versus placebo. The primary efficacy objective for Part B is to evaluate the proportion of participants treated with 1, 2.5, or 5 mg zibotentan combined with 10 mg dapagliflozin and 10 mg dapagliflozin monotherapy versus placebo achieving a ≥ 20% decrease in HVPG or a reduction to or below 12 mmHg in HVPG

This study aims to evaluate the efficacy and safety of lebrikizumab when used in combination with topical corticosteroid (TCS) treatment, compared with placebo, in pediatric participants with moderate-to-severe atopic dermatitis. Participants found to be eligible according to all of the study entry criteria will be randomly assigned in a 2:1 ratio to receive either lebrikizumab or placebo. This study can last up to 32 weeks, with 4 study periods. Screening Period: up to 4 weeks (≤30 days), TCS Standardization Period: 2 weeks, Treatment Period: 16 weeks, Post-Treatment Safety Follow-up Period: 12 weeks.

The purpose of this research study is to find out if AMDS is safe and effective in the treatment of acute dissection (sudden tear). For patients whose aortic anatomy is suitable for treatment with AMDS, the use of this device may promote healing of the aortic walls and possibly reduce the need for additional aortic surgeries. If conventional surgery, in combination with AMDS, is effective, it is believed that this could lead to improved aortic healing; an improvement in healing could reduce the risk of hospitalization and reoperation in the chest compared to conventional surgery alone.

This study includes a longitudinal mixed methods study of 360 YYA with diabetes in SC, CO, and WA conducted with SFS 1 participants. SFS 2 will have two parts: (1) an intense, longitudinal study consisting of two 14-day assessment periods spaced 9 months apart which will include CGM, EMA, accelerometry, and surveys in all participants (the EMA study); and (2) a concurrent events qualitative study with a subset of participants (30 T1D, 15 T2D) who will complete 13 one-on-one, semi-structured interviews throughout the 9-month assessment period (the qualitative study).

The need to advance knowledge and increase access to treatment for co-occurring posttraumatic stress disorder (PTSD) and substance use disorders (SUD) is a priority for service members and Veterans. PTSD and SUD often co-occur and are two of the most common and debilitating mental health disorders among service members and Veterans. However, few providers are trained in this approach and therefore are not prepared to deliver best practice care to Veterans who need integrated PTSD/SUD treatment.
This project will directly address this critical gap in provider training to improve the care of Veterans with comorbid PTSD/SUD, by providing development and testing of a web-based provider training course for concurrent treatment of PTSD and SUD Using prolonged exposure (COPE).

The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF pulmonary exacerbations during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean ppFEV1 (pulmonary function testing) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms. The study will compare treatment of an intravenous aminoglycoside and β-lactams (AG) versus intravenous β-lactams only (non-AG) in people with CF diagnosed with a pulmonary exacerbation who will be treated for Pa.

The purpose of this study is to develop and test if upper limb task practice and muscle activity training improve upper limb function in stroke survivors. Participants will be asked to come to the laboratory 3 times a week for 6 weeks to receive upper limb task practice and/or muscle activity training. Participants will also come to the laboratory for additional 3 visits for assessments of upper extremity function. The total duration of the study will be 2.5 months.

This study is for patients that have been diagnosed with locally advanced head and neck squamous cell carcinoma. The purpose of this research study is to evaluate the effectiveness of using a combination of pembrolizumab and olaparib when given before and after standard chemoradiation therapy in treating locally advanced head and neck squamous cell carcinoma. Pembrolizumab and olaparib are drugs that are approved for treatment of different cancers including lung, head and neck, breast and prostate cancer. However, FDA has not approved use of these two drugs together in treating head and neck cancer.

Treatment will be offered in three phases. In the induction phase, participants will receive a single infusion of pembrolizumab and will take olaparib tablets twice daily for total of 21 days. Participants will move to the chemoradiation phase, where they will receive radiation therapy and chemotherapy per routine standard care, for a total of 7 weeks. Chemoradiation therapy is done on a daily basis (excluding weekends), and chemotherapy therapy will involve a cisplatin infusion once weekly. At the conclusion of this phase, participants start the maintenance phase, which involves treatment with pembrolizumab and olaparib in cycles that are 42-days long. Treatment will include a single pembrolizumab infusion during each cycle and taking olaparib tablets twice daily during each cycle. Total number of cycles to be completed in the maintenance phase are 8 cycles. Participants can expect to be in this study for about 6.5 years.